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Latest News: Research

NHS England replies to the joint clinicians/charities letter

29 September 2017 / Posted in: Research

The request to include those with more than two SMN2 copies and over 7 months of age in the SMA Type 1 nusinersen EAP is not yet successful. We are still waiting to hear from NICE about next steps.

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Novartis Releases Clinical Trial Update

27 September 2017 / Posted in: Research

Novartis has released the following community update for the LMI070 (branaplam) clinical trial.

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Roche’s SUNFISH study community update

19 September 2017 / Posted in: Research

Roche have provided an update on clinical trials of their SMN2 splice-modifying drug RG7916

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Revisiting the Consensus Statement for Standards of Care in SMA - Full Report

30 August 2017 / Posted in: Research

You can now read the full workshop report written by the clinical researchers and patient representatives from Europe and the US who met last year.

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Letter to NHS England Requesting Review of EAP Type 1 Interim Policy Criteria

24 August 2017 / Posted in: Research

Sent on 23rd August from clinicians and advocacy groups, this asks for the age limit and SMN2 copy number restrictions on potential access to be removed.

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RG7916 FIREFISH trial now enrolling patients (though not in UK)

16 August 2017 / Posted in: Research

Roche has begun to enrol SMA patients in the FIREFISH Phase II trial of their splice-modifying drug, RG7916 (also known as RO7034067).

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Age of children who will be able to access the EAP for SMA Type 1 going forwards

07 August 2017 / Posted in: Research

We have clarified that, from now on going forwards (until we hear from NICE), children will have to be diagnosed by 7 months of age to be eligible. If you have a child who has already been diagnosed with SMA Type 1 who is older than this and not already on the EAP, please read more.

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NHS England Announces Interim Policy on EAP for SMA Type 1

04 August 2017 / Posted in: Research

All hospital costs for administering nusinersen to patients with SMA Type 1 will be provided by NHS England.

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Brief Report from the All Party Parliamentary Group that focused on nusinersen

21 July 2017 / Posted in: Research

Held on 18th July, this covered the current provision of nusinersen for infants with SMA Type 1 through the Expanded Access Programme (EAP) and what progress there has been towards the possibility of wider access.

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Biogen announces how long they intend to keep open UK EAP for SMA Type 1

13 July 2017 / Posted in: Research

They state that this will be for ‘the remainder of the year in the UK’ and that they will ‘re-evaluate the progress of the reimbursement progress and the EAP towards the end of 2017’.

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